Targeting ganglioneuromas with mTOR inhibitors.

We recently identified activated protein kinase B (PKB/AKT) as a tumorigenic driver in childhood ganglioneuroma. Inhibition of the mechanistic target of rapamycin (mTOR), a serine/threonine kinase downstream of AKT, effectively reduced the tumor burden in zebrafish with ganglioneuroma. We propose a clinical trial of mTOR inhibitors as a means to shrink large ganglioneuromas prior to surgical resection.